Active — Not RecruitingPhase 1

Phase Ib Clinical Study to Evaluate the Safety and Tolerability of VSA012 Injection in Paroxysmal Nocturnal Hemoglobinuria

China50 participantsStarted 2025-04-15

Plain-language summary

The complement system is an important component of the innate immune system. Abnormal activation, inadequate regulation and control of the complement system, as well as impaired and dysfunctional effector functions, underlie complement mediated diseases including PNH. VSA012 targeting complement system has the potential to treat a variety of diseases associated with abnormal activation of the complement system.The purpose of VSA012-1002 is to evaluate the safety, tolerability, pharmacokinetic, pharmacodynamics and efficacy of VSA012 Injection in subjects with PNH.

Who can participate

Age range18 Years – 75 Years

SexALL

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Exclusion criteria

✕. Having never received any complement inhibitor therapy;
✕. Having received C5, C3, or CFB complement inhibitors and having discontinued the complement inhibitor for more than 5 half-lives or 3 months prior to screening;
✕. Reticulocyte count \< 100 × 109/L;
✕. Platelet count \< 30 × 109/L;
✕. Neutrophil count \< 0.5 × 109/L;
✕. Creatinine clearance \< 30 mL/min (calculated by the Cockcroft-Gault formula);
✕. Reticulocyte count \< 100 × 109/L;
✕. Platelet count \< 30 × 109/L;

What they're measuring

Number of Participants with Treatment-Emergent Adverse Events (AEs) and/or Serious Adverse Events (SAEs)

Timeframe: up to Day 540

preliminary efficacy

Timeframe: up to Day 540

Trial details

NCT IDNCT06848296

SponsorBisirna Therapeutics Pte. Ltd.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2027-02-28

View on ClinicalTrials.gov More Paroxysmal Nocturnal Hemoglobinuria (PNH) trials